Azacitidine for Relapse After Allogeneic Stem Cell Transplantation-Single-Center Study

Area of Science:

• Hematology
• Oncology
• Stem Cell Transplantation

Background:

• Relapse remains a primary cause of treatment failure in myeloid malignancies post allogeneic hematopoietic stem cell transplantation (allo-HSCT).
• Limited therapeutic options exist for managing relapse, highlighting the need for effective strategies.

Purpose of the Study:

• To evaluate the efficacy and outcomes of azacitidine use in patients with myeloid malignancies undergoing or having undergone allo-HSCT.
• To compare the effectiveness of azacitidine when used preemptively versus for overt relapse or maintenance therapy.

Main Methods:

• A retrospective, single-institution study of 28 patients with myeloid malignancies treated with azacitidine.
• Patients received azacitidine for overt relapse, preemptive therapy, or post-transplant maintenance.
• Donor lymphocyte infusions (DLIs) were administered to some patients.

Main Results:

• Preemptive azacitidine therapy was associated with significantly longer median overall survival (21.2 months) compared to relapse therapy (6.1 months).
• In the preemptive group, 50% achieved complete cytogenetic remission and 14% had stable minimal residual disease.
• Toxicity was observed, including neutropenia (71%), thrombocytopenia (36%), and serious infections (36%) in the preemptive setting.

Conclusions:

• Azacitidine is most effective when used as a preemptive therapy to prevent relapse after allo-HSCT for myeloid malignancy.
• Using azacitidine for ongoing relapse may stabilize disease and facilitate a second allo-HSCT.